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How Science and Strategic Collaboration Led to a New, “Personalized” Cystic Fibrosis Treatment for Some Patients

Targeting a drug for small subgroups of patients is a new way to find effective therapies. This is often called personalized medicine, and it’s one of today’s most promising areas of new drug development.

Last year, FDA approved two important targeted medicines: Xalkori (crizotinib), a lung cancer drug that targets tumors with the abnormal ALK gene, and Zelboraf (vemurafenib), a drug to treat malignant melanomas that have a certain gene mutation. Both drugs were approved with companion diagnostic tests to identify if patients have a susceptible tumor.

Today, the FDA approved Kalydeco (ivacaftor) to treat a specific subgroup of patients with cystic fibrosis (CF). Cystic fibrosis is an inherited genetic disease that affects a person’s lungs and other organs and may lead to an early death.

What makes the availability of Kalydeco even more unique is that the drug’s developer, Vertex Pharmaceuticals, teamed up with the Cystic Fibrosis Foundation to develop and study the drug.

This success story began in 1989 when a team of researchers, including Francis Collins, now the director of the National Institutes of Health, discovered the gene that is involved in cystic fibrosis. This gene, known as CFTR, plays an important role in producing a protein that regulates the flow of salt and water out of the cells that line the cavities of the body. There are a number of different mutations that can cause the CFTR gene to produce a defective protein. This results in lung congestion and digestive problems.

Kalydeco targets a gene mutation that only occurs in about 4 percent of CF patients. Before using this medicine, doctors will test CF patients to determine whether they have this mutation (many CF patients have already been tested to understand what caused their CF). If the patient is a match, the drug may provide substantial benefits including improved lung function and weight gain.

Patients have played an important role in how new drugs are developed and studied since the HIV/AIDS activists in the 1980s and 1990s. But what the Cystic Fibrosis Foundation pioneered is a new form of patient power that some have called venture philanthropy. The Foundation helped with a portion of the drug’s development costs, provided researchers with useful insights about the CF patient population and helped in the recruitment of study participants – contributions that were critical to quickly bringing the innovative new therapy to patients.

The unique and mutually beneficial partnership that led to the approval of this new therapy for some CF patients serves as a great model for future drug development and patient group collaboration moving forward.

Here’s to innovation and continued cooperation and progress for patients!

Janet Woodcock, M.D., is the Director for FDA’s Center for Drug Evaluation and Research

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FDA's official blog brought to you from FDA's senior leadership and staff stationed at home and abroad - sharing news, background, announcements and other information about the work done at the FDA on behalf of the American public.